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White Paper

A New Era in the Therapeutic Journey: Gene Therapy as the Beacon for Rare Diseases

Introduction

Historically, rare diseases treatment has focused on treating or minimizing symptoms via regular doses of small molecular drugs or other therapeutics. However, the revival of gene therapy and recent advancements in the field have provided opportunities to correct the genetic abnormalities that lead to disease. There has been a significant rise in gene therapy-based clinical trials since the late 1990s with an increase to several thousand as of early 2020. As a novel therapeutic modality, in this article, we discuss the strategies and opportunities gene therapy presents for rare diseases.